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NCT04833894 | RECRUITING | Generalized Myasthenia Gravis

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children with Generalized Myasthenia Gravis
Sponsor:

argenx

Brief Summary:

The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)

Condition or disease

Generalized Myasthenia Gravis

Intervention/treatment

Efgartigimod IV

Phase

PHASE2

PHASE3

Study Type : INTERVENTIONAL
Estimated Enrollment : 12 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children from 2 to Less Than 18 Years of Age with Generalized Myasthenia Gravis
Actual Study Start Date : 2021-10-26
Estimated Primary Completion Date : 2027-03
Estimated Study Completion Date : 2027-03

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 2 Years to 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • 1. Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
  • 2. Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent.
  • 3. Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation
  • 4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
  • 5. Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening.
  • 6. Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants (\<15kg) historical values can be used).
  • 7. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
  • 1. Male participants: Male participants must agree to not donate sperm from of providing informed consent/assent until they have completed the trial.
  • 2. Female participants: Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered.
Exclusion Criteria
  • 1. Participants with MGFA class I, IVb, and V.
  • 2. Female adolescents of childbearing potential: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP.
  • 3. Has any of the following medical conditions
    • 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
    • 2. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.
    • 3. History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP. Participants with the following cancers can be included at any time: Adequately treated basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological findings of prostate cancer
    • 4. Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk
    • 4. Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
    • 5. A documented lack of clinical response to plasma exchange (PLEX).
    • 6. Received a live or live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.
    • 7. Received a thymectomy \<3 months before screening or 1 is planned to be performed during the trial period.
    • 8. The following results from these diagnostic assessments will be considered exclusionary
      • a. Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection; Hepatitis C virus (HCV) based on HCV antibody assay; Positive HIV serology at screening; Positive nasopharyngeal swab polymerase chain reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at screening.
      • 9. Using the following prior or concomitant therapies: Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of IMP, Use of any monoclonal antibody within the 6 months before the first dose of IMP, Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or PLEX within 4 weeks before screening.
      • 10. Total immunoglobulin (IgG) levels \<6 g/L below the lower limit of normal (LLN) according to the reference ranges of the central laboratory for participant by sex and age at screening.
      • 11. A known hypersensitivity reaction to efgartigimod or any of its excipients.
      • 12. Current participation in another interventional clinical trial or previous participation in an efgartigimod trial with at least 1 dose of IMP received.
      • 13. History (within 12 months of screening) of current alcohol, drug, or medication abuse as assessed by the investigator.
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children with Generalized Myasthenia Gravis

Location Details

NCT04833894

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, Illinois

Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

Chicago, Illinois, United States, 60611

RECRUITING

United States, North Carolina

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States, 27599

RECRUITING

United States, Virginia

University of Virginia (UVA) Health - Developmental Pediatrics Clinic

Charlottesville, Virginia, United States, 22903

WITHDRAWN

Austria,

Medical University of Vienna

Wien, Austria, 1090

RECRUITING

Belgium,

University Hospital Antwerp

Antwerp, Belgium, 2650

RECRUITING

Canada,

British Columbia Children's Hospital

Vancouver, Canada, V6h 3n1

RECRUITING

France,

AP -HM - Hospital of the Timone

Marseille, France, 13385

RECRUITING

France,

Public assistance Hospitals in Paris (AP-HP)-Necker-Enfants sick hospital

Paris, France, 75015

RECRUITING

Georgia,

Vian - M. Iashvili Children's Central Hospital

Tbilisi, Georgia, 121

RECRUITING

Georgia,

Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic

Tbilisi, Georgia, 159

RECRUITING

Germany,

Charite University Medicine Berlin - Campus Virchow Clinic - Social Pediatric Center

Berlin, Germany, 13353

NOT YET RECRUITING

Germany,

University Hospital Essen

Essen, Germany, 45147

RECRUITING

Italy,

Polyclinic Consortium University Hospital of Bari

Bari, Italy, 70124

RECRUITING

Italy,

University Hospital A. Meyer

Florence, Italy, 50139

RECRUITING

Italy,

Giannina Gaslini Hospital

Genova, Italy, 16147

RECRUITING

Netherlands,

Leiden University Medical Center

Leiden, Netherlands, 2333 for

RECRUITING

Poland, Pomeranian Voivodeship

University Clinical Center

Gdańsk, Pomeranian Voivodeship, Poland, 80-952

RECRUITING

Poland, Slaskie Voivodeship

Synapsis Multi -specialist medical clinic

Katowice, Slaskie Voivodeship, Poland, 40-123

RECRUITING

Poland,

Central Clinical Hospital - University Clinical Center in WUM

Warsaw, Poland, 02-097

RECRUITING

Spain,

Sant Joan de Deu Hospital

Esplugues de Llobregat, Spain, 08950

RECRUITING

Spain,

University Hospital I POLITECNIC THE FAITH OF VALENCIA

Valencia, Spain, 46026

RECRUITING

United Kingdom,

Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital

London, United Kingdom, Wc1n 3jh

RECRUITING

United Kingdom,

Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital

Manchester, United Kingdom, M13 9wl

RECRUITING

United Kingdom,

Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital

Oxford, United Kingdom, Kassah may